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The purpose of this study is to evaluate the long-term safety of intramuscular administration of Engensis in Participants with Amyotrophic Lateral Sclerosis who were previously randomized, received treatment, and completed the Day 180 Visit of Study VMALS-002-2. Safety will be assessed by incidences of treatment-emergent adverse events, treatment emergent serious adverse events, adverse events of special interest, and the clinically significant laboratory values.
VMALS-002-2b was a safety extension study following VMALS-002-2, which was a Phase 2a, double-blind, randomized, placebo-controlled, multicenter study designed to assess the safety of intramuscular administration of Engensis in subjects with Amyotrophic Lateral Sclerosis.Following completion of the Day 180 visit in VMALS-002-2 and signing of written informed consent, subjects were enrolled in this approximately 180-day extension study, with subjects completing combined participation with a Day 365 visit. Assessments to be conducted during this extension study were as follows: vital signs, weight and height, complete physical examination, 12-lead electrocardiogram, a record of all concomitant medications and procedures, clinical laboratory assessments, Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, handheld dynamometry, slow vital capacity, Amyotrophic Lateral Sclerosis Assessment Questionnaire, 40 questions, Patient Global Impression of Change, and Clinical Global Impression of Change. Assessments of treatment-emergent adverse events, treatment-emergent serious adverse events, adverse events of special interest, and clinically significant laboratory values continued from the Day 180 visit of Study VMALS-002-2 through the VMALS-002-2b extension study to Day 365.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Engensis | Active Comparator | Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart |
|
| Placebo | Placebo Comparator | Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Engensis | Biological | Lyophilized biologic to be reconstituted containing Engensis |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term | To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population) | Day 0 to Day 365 |
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| Measure | Description | Time Frame |
|---|---|---|
| To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants | Changes from Baseline (Study VMALS 002-2, Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating TOTAL scores at Day 365 for Engensis compared to Placebo There are 12 separate assessments in this neuromuscular assessment covering Bulbar, Fine Motor, Gross Motor and Breathing assessments. Each individual assessment is scored from 0 (worst case) to 4 (best case). Total score is calculated as the sum of all 12 assessment subscores. Note that only the Total Score will be reported in the clinical study report due to the small number of subjects. The Total Score range is from a minimum of 0 (worst case) to a maximum of 48 (best case). |
Inclusion Criteria:
Exclusion Criteria:
- None
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Austin Neuromuscular Center | Austin | Texas | 78759 | United States | ||
| Hanyang University Medical Center |
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| ID | Title | Description |
|---|---|---|
| FG000 | Engensis | Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis |
| FG001 | Placebo | Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Demographic and Baseline Characteristics (Safety Analysis Population)
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| ID | Title | Description |
|---|---|---|
| BG000 | Engensis | Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis |
| BG001 | Placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Treatment-Emergent Adverse Events in More Than 2 Subjects Overall by System Organ Class and Preferred Term | To evaluate the long-term safety of intramuscular injections of Engensis in Participants with Amyotrophic Lateral Sclerosis in more than 2 Participants by System Organ Class and Preferred Term (Safety Analysis Population) | Percentages were based on the number of subjects in the Safety Analysis Population by treatment group and overall. Subjects were counted once within for each unique Preferred Term. | Posted | Count of Participants | Participants | Day 0 to Day 365 |
|
Day 0 to Day 365, or until participant withdraws informed consent for this extension study, which can be anytime between Day 180 and Day 365.
All study-related Adverse Events will be collected starting after completion of the Informed Consent process at Screening to Day 0/randomization of the VMALS-002-2 study. Treatment-Emergent Adverse Events and Treatment-Emergent Serious Adverse Events will be collected after first injections of VMALS-002-2 study until the time that the Participant withdraws their informed consent for this extension study.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Engensis | Active Comparator: Engensis 64 mg Engensis per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Engensis: Lyophilized biologic to be reconstituted containing Engensis |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site bruising | General disorders | MeDRA 23.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jinsub Lee, PhD. | Helixmith Co., Ltd. | +82-10-8256-0439 | jinsub.lee@helixmith.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Oct 12, 2021 | Feb 1, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 30, 2023 | Feb 1, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Placebo |
| Other |
Injectable Liquid |
|
| Day 0 and Day 365 |
| To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants | As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities. Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 365 by Muscle Group (ITT Population) | Day 0 and Day 365 |
| To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants | Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation. It is measured in Liters of air and the percentage of change from baseline is the Percent Predicted Value and Change from Baseline. Change from Baseline (Study VMALS 002-2, Day 0) in Slow Vital Capacity on Day 240, Day 300 and Day 365 for Participants with intramuscular administration of Engensis compared to Placebo. Increasing negative Change from Baseline results indicates worsening of respiratory capacity. | Day 0, Day 240, Day 300 and Day 365 |
| To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy | Total participants requiring Tracheostomy procedures that received intramuscular administration of Engensis, compared to total Placebo participants requiring Tracheostomy procedures. | Day 0 to Day 365 |
| To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality | Total all-cause Mortality for participants that received intramuscular injections of Engensis, compared to total all-cause Mortality for Placebo participants. Total participant deaths that received intramuscular administration of Engensis, compared to total Placebo participant deaths. | Day 0 to Day 365 |
| To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The Participant completes the 40 questions in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case). Note for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms. The Total Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population. The Change from Baseline was calculated and displayed in the Table. ALSAQ-40 was completed at the pre-dose baseline visit (Day 0), and at the Day 240 and Day 365 visits. | Day 0, Day 240 and Day 365 |
| To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life. Each descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that as made all the difference. The test was self-administered on Days 240 and 365. | Day 240 and Day 365 |
| To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging rom Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 240 and 365/ Early Termination. | Day 240 and Day 365 |
| Seoul |
| 04763 |
| South Korea |
Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG001 | Placebo | Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid |
|
|
| Other Pre-specified | To Evaluate Changes in Muscle Function Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants | Changes from Baseline (Study VMALS 002-2, Day 0) in Revised Amyotrophic Lateral Sclerosis Function Rating TOTAL scores at Day 365 for Engensis compared to Placebo There are 12 separate assessments in this neuromuscular assessment covering Bulbar, Fine Motor, Gross Motor and Breathing assessments. Each individual assessment is scored from 0 (worst case) to 4 (best case). Total score is calculated as the sum of all 12 assessment subscores. Note that only the Total Score will be reported in the clinical study report due to the small number of subjects. The Total Score range is from a minimum of 0 (worst case) to a maximum of 48 (best case). | Revised Amyotrophic Lateral Sclerosis Function Rating Total Score and Change from Baseline to Day 365 (Safety Analysis Population) | Posted | Mean | Standard Deviation | score on a scale | Day 0 and Day 365 |
|
|
|
| Other Pre-specified | To Evaluate Muscle Strength Changes Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants | As assessed bilaterally by Hand-Held Dynamometry in muscles in the upper and lower extremities. Muscle Strength as Measured by Handheld Dynamometry (lbs.) and Change from Baseline to Day 365 by Muscle Group (ITT Population) | ITT Population | Posted | Mean | Standard Deviation | Pounds | Day 0 and Day 365 |
|
|
|
| Other Pre-specified | To Determine Whether Intramuscular Administration of Engensis Has Effects on Respiratory Capacity in Amyotrophic Lateral Sclerosis Participants | Slow vital capacity is a pulmonary function test that quantifies the volume of air that can be slowly exhaled after slow maximum inhalation. It is measured in Liters of air and the percentage of change from baseline is the Percent Predicted Value and Change from Baseline. Change from Baseline (Study VMALS 002-2, Day 0) in Slow Vital Capacity on Day 240, Day 300 and Day 365 for Participants with intramuscular administration of Engensis compared to Placebo. Increasing negative Change from Baseline results indicates worsening of respiratory capacity. | Slow Vital Capacity Percent Change from Baseline by Visit (ITT Population) | Posted | Mean | Standard Deviation | percentage of Change from Baseline | Day 0, Day 240, Day 300 and Day 365 |
|
|
|
| Other Pre-specified | To Determine if Intramuscular Engensis Has Effects on Respiratory Function in Amyotrophic Lateral Sclerosis Participants - Tracheostomy | Total participants requiring Tracheostomy procedures that received intramuscular administration of Engensis, compared to total Placebo participants requiring Tracheostomy procedures. | Total Tracheostomy procedures for the Safety Analysis Population - Number of Participants - Day 0 to Day 365 | Posted | Count of Participants | Participants | Day 0 to Day 365 |
|
|
|
| Other Pre-specified | To Determine if Intramuscular Administration of Engensis Has Positive Effects on Survival in Amyotrophic Lateral Sclerosis Participants - All-Cause Mortality | Total all-cause Mortality for participants that received intramuscular injections of Engensis, compared to total all-cause Mortality for Placebo participants. Total participant deaths that received intramuscular administration of Engensis, compared to total Placebo participant deaths. | Total all-cause Mortality for participants | Posted | Count of Participants | Participants | Day 0 to Day 365 |
|
|
|
| Other Pre-specified | To Evaluate Quality of Life Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The Participant completes the 40 questions in the Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) with 5 categories: Physical Mobility, Activities of Daily Living and Independence, Eating and Drinking, Communication, and Emotional Reactions. Each question has 5 responses to select from: 0-Never (Best Case), 1-Rarely, 2-Sometime, 3-Often, and 4-Always (Worst Case). Note for each question there is a Minimum of 0 (Best), to the Maximum 4 (Worst). Decreasing scores indicates improvement of symptoms. The Total Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) scores are per Category as percentages of 100% maximum. Each Category percentage is summed for the Intent-to-treat Population. The Change from Baseline was calculated and displayed in the Table. ALSAQ-40 was completed at the pre-dose baseline visit (Day 0), and at the Day 240 and Day 365 visits. | Percent Change from Baseline (ITT Population) for Engensis and Placebo. | Posted | Mean | Standard Deviation | percentage of Change from Baseline | Day 0, Day 240 and Day 365 |
|
|
|
| Other Pre-specified | To Evaluate Patient Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The subject's impression of change after treatment was measured with the Patient Global Impression of Change questionnaire through use of the electronic Patient Reported Outcome . This questionnaire measures the subject's perception of how treatment has affected their level of activity, symptoms, emotions, and overall quality of life. Each descriptor is ranked on an increasing improvement scale; where 1 = No change (or condition has got worse), 2=Almost the same, hardly any change at all, 3=A little better, but no noticeable change, 4=Somewhat better, but the change has not made any real difference, 5=Moderately better, and a slight but noticeable change, 6=Better, and a definite improvement that has made a real and worthwhile difference, and 7 = A great deal better, and a considerable improvement that as made all the difference. The test was self-administered on Days 240 and 365. | Patient Global Impression of Change - ITT Population. | Posted | Count of Participants | Participants | Day 240 and Day 365 |
|
|
|
| Other Pre-specified | To Evaluate Clinician Reported Outcome Improvement Following Engensis Injections in Amyotrophic Lateral Sclerosis Participants Compared to Placebo | The Clinical Global Impression of Change is a validated instrument completed by observers as an assessment of Quality of Life. The Clinical Global Impression of Change is an 8-point scale with scores ranging rom Marked Improvement, Moderate Improvement, Minimal Improvement, Slight Improvement, and Unchanged (or Worse), along with an efficacy index with questions in a matrix for therapeutic effect and side effects.The test was completed on Days 240 and 365/ Early Termination. | Clinical Global Impression of Change - ITT Population | Posted | Count of Participants | Participants | Day 240 and Day 365 |
|
|
|
| 0 |
| 4 |
| 1 |
| 4 |
| 4 |
| 4 |
| EG001 | Placebo | Placebo Comparator: Placebo 32 mL of Placebo per Treatment Cycle, with each of 3 cycles composed of 2 days of 128 injections each to the right and left target muscles, spaced 2 weeks apart Placebo: Injectable Liquid | 0 | 4 | 1 | 4 | 4 | 4 |
| Peripheral arterialocclusive disease | Vascular disorders | MeDRA 23.0 | Systematic Assessment |
|
| Asthenia | General disorders | MeDRA 23.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MeDRA 23.0 | Systematic Assessment |
|
| Fatigue | General disorders | MeDRA 23.0 | Systematic Assessment |
|
| Injection site pain | General disorders | MeDRA 23.0 | Systematic Assessment |
|
| Injection site reaction | General disorders | MeDRA 23.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Productive cough | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Oropharyneal pain | Respiratory, thoracic and mediastinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MeDRA 23.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MeDRA 23.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MeDRA 23.0 | Systematic Assessment |
|
| Haemorrhoids thrombosed | Gastrointestinal disorders | MeDRA 23.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MeDRA 23.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| Muscle twitching | Musculoskeletal and connective tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MeDRA 23.0 | Systematic Assessment |
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| Kidney infection | Infections and infestations | MeDRA 23.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MeDRA 23.0 | Systematic Assessment |
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| Dysarthria | Nervous system disorders | MeDRA 23.0 | Systematic Assessment |
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| Muscle contractions involuntary | Nervous system disorders | MeDRA 23.0 | Systematic Assessment |
|
| Paraesthesia | Nervous system disorders | MeDRA 23.0 | Systematic Assessment |
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| Sinus tachycardia | Cardiac disorders | MeDRA 23.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MeDRA 23.0 | Systematic Assessment |
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| Multiple injuries | Injury, poisoning and procedural complications | MeDRA 23.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| Skin irritation | Skin and subcutaneous tissue disorders | MeDRA 23.0 | Systematic Assessment |
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| Hypertension | Vascular disorders | MeDRA 23.0 | Systematic Assessment |
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| Peripheral arterial occlusive disease | Vascular disorders | MeDRA 23.0 | Systematic Assessment |
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| Tachycardia | Vascular disorders | MeDRA 23.0 | Systematic Assessment |
|
| Tinnitus | Ear and labyrinth disorders | MeDRA 23.0 | Systematic Assessment |
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| Bone density descreased | Investigations | MeDRA 23.0 | Systematic Assessment |
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| Hypophagia | Metabolism and nutrition disorders | MeDRA 23.0 | Systematic Assessment |
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| Urinary retention | Renal and urinary disorders | MeDRA 23.0 | Systematic Assessment |
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| Dysmenorrhoea | Reproductive system and breast disorders | MeDRA 23.0 | Systematic Assessment |
|
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| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Shoulder Flexion- Right - Baseline - Actual |
|
| Shoulder Flexion-Right - Day 365 - Change from Baseline |
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| Elbow Flexion- Left - Baseline - Actual |
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| Elbow Flexion-Left - Day 365 - Change from Baseline |
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| Elbow Flexion- Right - Baseline - Actual |
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| Elbow Flexion-Right - Day 365 - Change from Baseline |
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| Day 365/Early Term - Change from Baseline |
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| Day 240 - Activities of Daily Living/Independence |
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| Day 365/Early Termination - Activities of Daily Living/Independence |
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| Day 240 - Eating and Drinking |
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| Day 365/Early Termination - Eating and Drinking |
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| Day 240 - Communication |
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| Day 365/Early Termination - Communication |
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| Day 240 - Emotional Functioning |
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| Day 365/Early Termination - Emotional Functioning |
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| Day 240 - Impression change - Almost the same, hardly any change |
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| Day 365/Early termination - Impression change - or worse |
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| Day 365/Early Termination - Impression change - Almost the same, hardly any change |
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| Day 240 - Minimally worse |
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| Day 240 - Much worse |
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| Day 365 / Early Termination - Minimally improved |
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| Day 365 / Early Termination - No change |
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| Day 365 / Early Termination Minimally worse |
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| Day 365 / Early Termination Much worse |
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